Biohaven Advances Toward Potential First FDA-Approved Therapy For Spinocerebellar Ataxia, FDA Drops Advisory Meeting

Biohaven Ltd. (NYSE:BHVN) stock is trading higher on Friday following an update with respect to its New Drug Application for troriluzole for adult patients with Spinocerebellar Ataxia (SCA).

In May 2025, the Division of Neurology 1 within the Office of Neuroscience of the Food and Drug Administration (FDA) informed the company that it was extending the Prescription Drug User Free Act (PDUFA) date for the troriluzole NDA by three months to provide time for a full review of the company’s recent data submissions.

The Division also informed Biohaven that it was planning to hold an advisory committee meeting to discuss the application.

Also Read: Strong Immunoglobulin Reduction Data Positions Biohaven’s BHV-1300 As Potential Autoimmune Therapy

In an SEC filing, the Division communicated that it had determined that an advisory committee meeting is no longer needed for regulatory decision-making.

The FDA’s decision regarding the NDA is expected during the fourth quarter of 2025.

If approved, troriluzole would be the first and only FDA-approved treatment for SCA. Spinocerebellar ataxia is a group of dominantly inherited neurodegenerative disorders characterized by progressive loss of voluntary motor control and atrophy of the cerebellum and brainstem. SCA affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom.

In April, Biohaven announced an up to $600 million non-dilutive capital agreement with Oberland Capital Management LLC. The agreement, with $250 million in gross proceeds received on closing on April 30, 2025, is expected to support commercial launch planning in SCA, clinical development activities, and ongoing business operations.

In September 2024, Biohaven released topline results from pivotal Study BHV4157-206-RWE demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after three years of treatment.

The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2.

Investors should note that in June, the FDA decided not to hold an Advisory Committee meeting for Capricor Therapeutics Inc.’s (NASDAQ:CAPR) Deramiocel, the company’s lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD).

DMD, like SCA, is a severe, progressive genetic disorder that causes muscle weakness and degeneration, primarily affecting boys.

Price Action: BHVN stock is trading higher by 11.65% to $16.40 premarket at last check Friday.

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